Week Note 2016 - 18

Week of: 

September 19, 2016


And (free?) data for all


This Friday, the department of Health and Human Services released an (unpublished) version of rules that both clarify and enforce data sharing for clinical trials through guidelines for registration of trials as well as submitting summary results. NIH also concurrently released a policy on the dissemination of NIH-funded trials through clinicaltrials.gov. Both these rules have been in the works for a while, and have been released after an extensive comment period.


As already picked up by several news outlets, the rules are expected to lead to more information being available on clinical trials to everyone, including patients. Notable is the requirement to share data on trials that failed, something we have talked about in previous weeknotes. The guidelines have clarified a number of issues, including which trials it applies to, submission of results stratified by ethnic and racial groups, and the protocol and statistical methods used. The NIH has also outlined further initiatives, including requiring good clinical practice (GCP) training, suggesting the use of a standardized NIH-FDA protocol, and development of a standardized electronic system for NIH to manage all these trials.


Of course, over the past year and a half, we have been saying that making this world less “opaque,” as Rob Califf put it recently, is essential. But implementing the idea is often more important than the idea itself. As we move our project from idea generation and synthesis towards developing a portfolio of recommendations, we grapple with a number of issues that occur whenever good ideas are implemented. These issues are important, because while a top-down initiative like this can cause change, complementary efforts can often lead to smoother and quicker transitions to improve system function.


First, any implementation of a plan like this absolutely requires stakeholder buy-in. Over the past year, we have heard a prolific trialist say “You call it data sharing, I call it blackmail.” We’ve heard trial sites say that putting data in databases like clinicaltrials.gov is such a pain that often they put in the minimum required information to put in the format, which doesn’t even get to the salient points of the trial. Curt Meinert has released a number of memos that outline the concerns of many people actually doing trials. And this is just at the level of conducting the trial. Even at higher levels, following these rules properly will require trained individuals who can understand and aggregate the data, statisticians throughout the trial development process, and maybe even a change in how companies and universities do trials. The bigger question is how this will be enforced, since currently there are no extra funds available to HHS for enforcement. Rob Califf believes that it won’t be an issue, but given the previous problems we have had with reporting results from trials, and the fact that this new rule essentially forces people to share data using a system they are not happy with, one of the jobs of our team will be to think of complementary solutions that can help move this great idea of sharing information forward.


So what could be an answer to align stakeholders? That brings us to our second point, stakeholders need to know how certain policies can benefit them in the long run, and what will be done with their data in the short run. In general, is there any evidence available that sharing such data from clinical trials will vastly improve the system? Are there quantifiable impacts that can be put forward to show how these initiatives can be better for everyone? And this is where we can start thinking about complementary efforts to help the system. For example, we have developed a machine learning model that predicts success of trials using data on trials from two different databases. Other groups have worked on similar models for more targeted diseases and populations. Efforts like these show the clear advantages of data sharing, give examples to stakeholders on how the data might be used and what potential benefits can be achieved, and turn a top-down policy into a system-wide solution.


Which brings us to our third point. How exactly does HHS and NIH anticipate these actions will change the clinical trials system? In many other systems, a solution like this just doesn’t change one aspect of data sharing, it always has system-wide ramifications. These can be positive, where it encourages all stakeholders to work together and be more open about their findings. Or it could be negative, where we see stakeholders finding ways around the policy to share even less. Even at the micro-level, what ramifications does this have for the patient as they sign to be on a trial, the CRA who is actually conducting the trial, and us as the population who need the drugs? We have been working with the AACT database from clinicaltrials.gov for over a year now, and from the population’s persepctive, the problem isn’t that there are not enough trials on there. The major issue is that it is difficult to navigate, and almost impossible to understand the information.


And that, we feel, is the most important issue here. Drawing an impact line from HHS to the stakeholders in the clinical trials system to the patients. How can we leverage this rule to really help patients? More importantly, will the new equilibrium in the system created by this rule impact the patient negatively? We have to make sure that good ideas and initiatives are not lost. Developing system-wide solutions that can complement progressive efforts by the government is the best way to go.


Week Notes Authors

Johns Hopkins University Co-Research Lead

Co-Research Lead

Jen Bernstein has 15 years experience working on WHO-, industry-, U.S. government-funded research studies and clinical trials.  She received her Master of Public Health at the Johns Hopkins Bloomberg School of Public Health and currently works as Part-Time Faculty in the Johns Hopkins Whiting School of Engineering. Jen is also a clinical research associate and independent research consultant providing research support for deliverables at all stages of project development including IRB submissions, journal articles, presentations, and grant submissions. Jen honed her skills at a variety of institutions including The Urban Institute, the Johns Hopkins Center for Communication Programs, and The Weinberg Center for Women's Health and Medicine at Mercy Medical Center. 

MIT Collaborative Initiatives

Ellie Carlough is currently Associate Director of MIT Collaborative Initiatives (MIT-CI) and has been with the organization since September 2007.  In this role, she is actively involved in project management, writing and strategic planning for MIT-CI, which applies a system-based approach to addressing widespread societal issues reaching a crisis point.

Ellie graduated from Mount Holyoke College and earned a Masters of Science in Non-profit Management from the New School for Social Research in New York, New York.  After working in Manhattan for 10 years in banking and most recently as Director of Corporate and Foundation Relations at St. Vincent’s Hospital and Medical Center of New York, Ms. Carlough spent a time in the challenging position of full-time mother mastering the art of multi-tasking and social networking before joining MIT-CI.

SITRA, MIT Collaborative Initiatives

Based in Boston, Justin is the Finnish Innovation Fund's (Sitra) academic liaison for North America and serves as Sitra's Senior Lead for Sustainable Development. He helped design and build the Low2No urban decarbonization initiative and manage its implementation, and served as Helsinki Design Lab's expert on energy, carbon and sustainability. 

Currently he is helping Sitra better align its endowment with its mission by developing ways to place capital in businesses, projects and funds that generate measurable social and environmental impact together with financial return. As part of Sitra’s Strategic Research team, he works to support and challenge Sitra's strategy and shared assumptions. His research includes developing a more comprehensive understanding of sustainable well-being and how Nordic countries can transition toward it with practical economic and public policy measures.

Justin is also an Adjunct Professor at the Rhode Island School of Design where he teaches graduate level design courses. He holds a Master of Architecture degree from the Harvard Graduate School of Design and a Bachelor of Arts from the University of Washington. Find him @justinwcook.

Johns Hopkins University

Viva Dadwal is a Visiting Scholar at Johns Hopkins University Bloomberg School of Public Health, where she is investigating the transfer of health innovations from low to high-income countries. She is a published academic, a Senior Fellow at the Centre on Governance at the University of Ottawa and a Deputy Editor of open-access global health journal, Globalization and Health. Previously, she worked as a Trade Policy Officer for Foreign Affairs, Trade and Development Canada. Viva has held prestigious internships at the World Health Organization and Permanent Mission of Canada to the United Nations and to the World Trade Organization in Geneva, Switzerland.

In her spare time, Viva promotes public astronomy in urban cities through a project called #popscope. She also guest blogs for World Bank’s youth blog, Youthink! Viva holds a B.Sc. (Hons) in Biology from the University of Windsor and an M.A. in Public and International Affairs from the University of Ottawa. Viva is a recipient of Fulbright Canada Award 2014-2015 and a member of the World Economic Forum Global Shapers community. Follow Viva's adventures on Twitter @vivadadwal.

Johns Hopkins University

Francisco Del Canto Viterale is Postdoctoral Researcher in the Systems Institute within the Whiting School of Engineering at Johns Hopkins University. Currently, he is working on two different research projects: “Complex International Innovation Partnerships”, and “Clinical Trials Systems”.

His core area of expertise is the intersection of Science Technology Innovation and Higher Education, and International Relations.

Francisco has been working in the academia for over seventeen years in several countries. He has an extensive experience working as a university lecturer in International Studies, International Migration and Latin American Studies; and also as a researcher in topics related to International Scientific Relations, Science and Innovation Policy and Interdisciplinary, Complex and Systems Approach.

He received his Ph.D. in International and Intercultural Studies from the University of Deusto (Spain) in January 2014. Prior to his doctorate, he earned a Bachelor of Science in International Relations (1998), a Diplomat in International Affairs and Diplomacy (1999) in Argentina, and a Masters in International Migrations and Social Cohesion (2011) in Spain.

Johns Hopkins University

Felipe Feijoo is a Postdoctoral researcher at the Systems Institute in the Whiting School of Engineering, The Johns Hopkins University.

He obtained his B.S. in Engineering Sciences at the Universidad de La Frontera, Chile. Thereafter, he pursued his Master degree (2011) and Ph.D. (2015) in Industrial Engineering at the University of South Florida, Tampa.  

His current research involves the application of stochastic complementarity models and economics to analyze the opening of clinical trials and the pharmaceutical market in the U.S. 

His research also focuses on the application of mathematical programming, game theory, and statistics for decision making in sustainable electricity markets. He worked on a mathematical-statistical framework to developed Pareto designs of cap-and-trade carbon policies with high penetration of green energy via micro-grids. Other research interests include health-economics and predicting-forecast modeling using machine learning techniques. Felipe has also worked and led several projects for the Tampa Electric Company (TECO), Florida.

Johns Hopkins University

Senior Advisor

Dr. Tak Igusa is a professor at the Johns Hopkins University who has been engaged in multi-disciplinary collaborations spanning a wide range of fields.  He currently holds appointments in the Departments of Civil Engineering, International Health, Earth and Planetary Sciences, and Applied Mathematics & Statistics.  To provide rigor to his collaborative activities, Dr. Igusa has been promoting systems science and methodologies, which has led to significant, new research programs within the university.  This includes projects funded by the NIH, Robert Wood Johnson Foundation, CDC and the National Science Foundation on systemic studies of childhood obesity, rheumatic disease, community resilience to natural disasters, and technology innovation in developing nations.  His work led to the establishment of the Johns Hopkins Systems Institute in 2011.

Dr. Igusa received an A.B. in Applied Mathematics from Harvard University and a PhD in Civil Engineering from University of California, Berkeley. At Johns Hopkins, he is currently the Director for Health of the Systems Institute and the Program Lead of the Education & Training Program at the Global Obesity Prevention Center.  He is also advising the development of systems research programs that bridge public health and engineering at the University of the Witwatersrand in Johannesburg, South Africa. 


Johns Hopkins University

Gary Lin is currently working on complex systems research with the Johns Hopkins Systems Institute and pursuing a Doctor of Philosophy in Civil Engineering at the Johns Hopkins University.  He has a variety of research interests which includes healthcare systems, infrastructure systems, supply chain and transportation networks, and human population dynamics.  Using his diverse background, Gary has been involved in numerous interdisciplinary projects that range from natural gas networks to environmental impacts of population growth.  He hopes to apply the systems approach towards large-scale, health and engineering issues that face present and future society.

Gary obtained his Bachelor of Science in Civil Engineering and Bachelor of Arts in Economics from the University of Colorado at Boulder.  He is also a registered Engineer-in-Training (EIT) in the state of Colorado and student member of the American Society of Civil Engineers.  In addition, Gary has experience working in food transportation logistics, financial administration, and construction marketing.  Gary enjoys disc golfing, cycling, and hiking in his spare time.

Johns Hopkins University

Paul Locke, an environmental health scientist and attorney, is an Associate Professor at the Johns Hopkins Bloomberg School of Public Health in the Department of Environmental Health Sciences.  He holds a joint appointment in the Department of Health Policy and Management.  Dr. Locke is also the Distinguished Visiting Professor of Animal Law and Science at the Northwestern School of Law of Lewis & Clark College.   Dr. Locke has an MPH from Yale University School of Medicine, a DrPH from the Johns Hopkins University Bloomberg School of Public Health and a JD degree from Vanderbilt University School of Law. 

At Hopkins, Dr. Locke leads an integrated public health research, practice and teaching program.  His program focuses on research-to-practice initiatives to better protect public health, especially in advancing evidence-based in vitro toxicology and radiation protection policy at federal and international organizations.  He has published widely in both law reviews and scientific and policy journals, and has developed three cross-disciplinary courses in environmental law and policy and animal law.  Dr. Locke also directs the School’s Doctor of Public Health program in Environmental Health Sciences and a certificate program in Humane Sciences and Toxicology Policy.

As a member of the clinical trials initiative team, Dr. Locke is helping to better understand the laws, regulations and policies that frame clinical trials and human subjects research, and seeking opportunities to utilize rapidly evolving in vitro toxicological methodologies and other new techniques in clinical trial decision-making.      

Dr. Locke is admitted to practice law in the state of New York and the District of Columbia, the Southern District Court of New York and is a member of the bar of the United States Supreme Court.

Muchieast, Rhode Island School of Design

Systems Innovation Design Lead

Enrique is the systems innovation and design lead for the project. He is the founder and director of Muchieast LLC, an international consultancy working at the intersection of social, cultural and urban contemporary practices by incorporating the principles of systems innovation and creative thinking. He is a Visiting Lecturer at Brown University and a Senior Critic at the Rhode Island School of Design. Enrique holds a Master in Architecture from the Universidad Politécnica de Madrid, Escuela Técnica Superior de Arquitectura de Madrid; and a Master in Industrial Design, with Honors, from the Rhode Island School of Design, where he was granted the Norman Bel Geddes award in 1998.

Johns Hopkins University, Bloomberg School of Public Health

Michele is currently a Senior Research Assistant at Johns Hopkins Bloomberg School of Public Health in the Department of Environmental Health Sciences. Her interests include risk management and communication, effective translation of science into policy, and more recently, using a systems approach to fix ineffective or outdated policy. Since obtaining a Master’s in Health Science and a Certificate in Risk Science and Public Policy in 2012, she has been working on communications with congressional offices on Capitol Hill, conducting legislative analysis, producing advocacy materials and organizing consultation meetings with congressional offices.

Johns Hopkins University Co-Research Lead

Co-Research Lead

Dr. Sauleh Siddiqui is an Assistant Professor of Civil Engineering with an appointment in AppliedMathematics & Statistics at Johns Hopkins University and is affiliated with the Johns Hopkins Systems Institute. He is also a Visiting Researcher and Lecturer at the Technical University of Berlin and the German Economic Research Institute (DIW Berlin). His research is on formulating and solving optimization and game theory models applicable to large-scale systems. Such systems arise when modeling problems in energy and environmental markets, public health, and transportation. He also models engineering design and develops novel algorithms along with supporting mathematical theory.

He has received research funding from the World Bank, Johns Hopkins Health System, the Environment Energy Sustainability Health Institute, and the Norwegian Research Council. In addition, he has collaborated on projects with the International Council on Clean Transportation, Institute of Medicine, National Academy of Engineering, and Bikemore. These projects have included modeling energy and climate markets, patient flow and staffing in hospitals, global vaccination strategies, and urban transport networks.       

He received an A.B. in Mathematics and Public Policy from Franklin & Marshall College and a Ph.D. in Applied Mathematics & Statistics, and Scientific Computation from the University of Maryland, College Park.